Is Nomlabofusp legal in the United States?

Human frataxin fused to a TAT derived cell penetrating peptide, injected subcutaneously to replace the protein missing in Friedreich ataxia. In 55 adults across two phase 1 trials it raised frataxin in buccal cells, skin and platelets; whether that changes the disease is the question of a 150 person phase 3 that began in August 2026 and reads out in 2029.

Friedreich ataxia is caused by too little frataxin, a mitochondrial protein, and nomlabofusp is an attempt to put the protein back. It is recombinant human frataxin joined to a cell penetrating peptide derived from HIV TAT, designed so the fusion crosses cell membranes and the mitochondrial import machinery cleaves it to release mature frataxin inside the mitochondrion.

Mechanism: In people, what is established is that subcutaneous nomlabofusp is absorbed quickly, that daily dosing raises frataxin measurably in buccal cells, skin and platelets, and that levels in those tissues correlate with each other. Whether frataxin reaches the tissues that matter most in Friedreich ataxia, the dorsal root ganglia, cerebellum and heart, in a person, has not been measured directly; the cross tissue correlations that support it come from animals.

Regulatory history: Not FDA approved. Phase 2 open label recruiting; phase 3 (NCT07778836, 150 participants) started 5 August 2026 with primary completion projected July 2029.

Approves prescription drugs by indication and polices unapproved products, including peptides sold online. Compounding pharmacies may prepare some compounds under a prescription when the FDA places them on a permitted bulk-substances category.

The classification above is the The Longevity Archive reading of U.S. Food and Drug Administration listings for Nomlabofusp at the reviewed date, not a legal opinion. Rules change, and importing, prescribing and possessing a compound can each be treated differently. Check the regulator directly before acting on anything here.